ASH virtual event hears about CRISPR and CAR-T based approaches to hard-to-treat blood disorders and cancers – BioPharma-Reporter.com

ASH virtual event hears about CRISPR and CAR-T based approaches to hard-to-treat blood disorders and cancers – BioPharma-Reporter.com

In the first study, researchers used CRISPR/Cas9 to treat two inherited blood disorders, beta thalassemia and sickle cell disease (SCD). The trial, which demonstrated remarkable improvements in all participants, is the first time this revolutionary approach has been used successfully in these patient populations.

“Given that the only FDA-approved cure for sickle cell disease, a bone marrow transplant, is not widely accessible, having another curative option would be life-changing for a large number of the sickle cell disease population,”​ said press briefing moderator, Dr Catherine Bollard, of Children’s National Research Institute and George Washington University. “While longer follow-up data are needed, this study is extremely exciting for the field.”​

The study – CRISPR-based gene editing ​

Investigators reported interim safety and efficacy data from 10 patients who received an investigational gene-editing based therapy, CTX001. The trials are the first to test a CRISPR-Cas9 gene editing therapy in humans for a genetic disease, the researchers reported.

Sickle cell disease (SCD) can cause a variety of health problems including episodes of severe pain, called vaso-occlusive crises, as well as organ damage and strokes, while patients with transfusion-dependent thalassemia (TDT) are dependent on blood transfusions from early childhood. The only available cure for both diseases is a bone marrow transplant from a closely related donor, an option that is not available for the vast majority of patients because of difficulty locating matched donors, the cost, and the risk of complications.

Published at Mon, 07 Dec 2020 16:17:29 +0000